Feature

Sarepta: High-Profile Decision for the FDA

Shares of orphan drug maker Sarepta could soar if the FDA gives an early go-ahead to its drug for Duchenne muscular dystrophy.

The U.S. Food and Drug Administration is facing growing pressure from patient advocates, medical experts, and members of Congress to approve an experimental drug from Sarepta Therapeutics for Duchenne muscular dystrophy, a fatal inherited muscle-wasting disease affecting children. The situation involving the drug, eteplirsen, is shaping up as one of the highest-profile drug decisions by the FDA since several AIDS treatments were approved in the 1980s and 1990s after strong public pressure from AIDS patients.